Author: Elizabeth Roman

Have you ever thought about joining a clinical trial that is trying to find out if an investigational drug works in treating your disease or medical condition?  Or maybe your healthcare provider has talked to you about treating you with an investigational drug through expanded access. 

An investigational drug can also be called an experimental drug and is being studied to see if your disease or medical condition improves while taking it. Scientists are trying to prove in clinical trials:

• If the drug is safe and effective.
• How the drug might be used in that disease.
• How much of the drug is needed.
• Information about the potential benefits and risks of taking the drug.

Not every person’s disease or medical condition responds the same way to approved drugs. Your healthcare provider might have talked to you about using an investigational drug if you have:

• Experienced side effects that are too severe to continue taking
• Limited treatment options available.
• Heard about promising early study results for a specific investigational drug.
• No approved drugs available to treat your disease or medical condition.

If your healthcare provider thinks that using an investigational drug is an option for you, then consider asking questions like those listed below before deciding whether it is right for you.

• What is this drug being studied for?
• How much is already known about the investigational drug?
• What basis is there for thinking that the drug will work better for your medical condition than using an approved treatment?
• What scientific evidence is available to support the use of this investigational drug?
• What are the potential risks of using this investigational drug?
• Are there other drugs that are already approved to treat your disease or condition?
• Have you already tried them?